WASHINGTON, April 22, 2016 (GLOBE NEWSWIRE) -- In what is predicted to be the largest turnout in history at an FDA Advisory Committee meeting, more than 800 clinicians, researchers, patients and families from around the world will attend a U.S. Food and Drug Administration (FDA) Advisory Committee Meeting (Ad Comm) on Monday, April 25, to urge the FDA to acknowledge positive clinical trial data and input from respected experts as the FDA considers approval of the Duchenne muscular dystrophy (DMD) drug eteplirsen.
Dozens of Duchenne patients, families, and scientists who have studied the disease and treated patients for decades will testify during the open public hearing section of the meeting, presenting compelling evidence of the drug’s efficacy and safety.
The Advisory Committee is an independent panel convened specifically to hear testimony in order to give guidance to the FDA as it considers approval of eteplirsen to treat Duchenne muscular dystrophy. DMD is a deadly muscle-wasting disease that affects one in approximately 3,500 boys worldwide. The disease is 100% fatal and there is no approved treatment in the United States.
The meeting comes two years after The Race to Yes, a community-driven initiative, gathered more than 100,000 petition signatures to send to the White House asking for the FDA’s expeditious review and approval of safe and effective treatments for Duchenne. As a result, the FDA pledged to apply “the use of all potential pathways” for DMD treatments.
The primary data under consideration in eteplirsen’s application are from a study of 12 patients who have been treated with the drug for five years. In a letter to the FDA, 36 Duchenne experts stated, “The collective signatories note that the group of 12 eteplirsen treated boys….is clearly performing better than our collective clinical experience and the published literature would predict. Collectively, a portion of us represent a group of physicians who have observed over 5,000 DMD patients in our practices over an average of more than 15 years.”
“The data demonstrate that eteplirsen is safe and effective,” said Race to Yes co-founder Tracy Seckler of Charley’s Fund. “We welcome a rigorous FDA review, and we expect an unbiased, thorough analysis based on sound science that meaningfully incorporates the perspective of experts in the field and patients who live with the disease.”
“Through the Food and Drug Administration Safety and Innovation Act (FDASIA), Congress gave the FDA the tools to apply flexibility in the evaluation of therapies for a life-threatening illness in order to accelerate approval to treat an unmet medical need,” said Race to Yes co-founder Marissa Penrod of Team Joseph. “Congress also urged the FDA to prioritize the patients’ perspective in evaluating new drugs and treatments. Our children are running out of time. We expect the FDA to honor FDASIA and fully employ the tools given to them by Congress.”
The #MakeDuchenneHistory coalition includes the Center for Duchenne Muscular Dystrophy at UCLA, the Duchenne Alliance, Jett Foundation, Parent Project Muscular Dystrophy, together with The Race to Yes.
About The Race to Yes
The Race to Yes is a community-driven initiative that mobilizes people who support rapid regulatory action to advance safe and effective treatments for Duchenne muscular dystrophy and amplifies their collective voice as a mechanism for change. We represent people who believe all stakeholders in the regulatory system have an obligation to urgently and proactively speed safe and effective treatments to patients. For more information, please visit www.theracetoyes.org